bomtabegagene bavoparvovec

Bomtabegagene bavoparvovec is an investigational gene therapy that uses an adeno-associated viral (AAV9) vector to deliver a functional copy of the *GLB1* gene to patients with GM1 gangliosidosis, a rare lysosomal storage disorder. The therapy aims to restore β-galactosidase enzyme activity by enabling cells to produce functional GLB1 protein. It was developed for intravenous administration and targeted at pediatric populations with GM1 gangliosidosis. The drug received orphan drug status and rare pediatric disease designation in the United States but its development was discontinued after Phase 1/2 clinical trials[2][4][7].

Brand names

AXO-AAV-GM1AXO-AAV-GM-1AXO-AAV-GM 1

Other names

bomtabegagene bavoparvovec